Browsing by Author "Macklin, Eric A."

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    A randomized controlled trial of an online health tool about Down syndrome
    (Elsevier, 2021) Chung, Jeanhee; Donelan, Karen; Macklin, Eric A.; Schwartz, Alison; Elsharkawi, Ibrahim; Torres, Amy; Hsieh, Yichuan Grace; Parker, Holly; Lorenz, Stephen; Patsiogiannis, Vasiliki; Santoro, Stephanie L.; Wylie, Mark; Clarke, Lloyd; Estey, Greg; Baker, Sandra; Bauer, Patricia E.; Bull, Marilyn; Chicoine, Brian; Cullen, Sarah; Frey-Vogel, Ariel; Gallagher, Maureen; Hasan, Reem; Lamb, Ashley; Majewski, Lisa; Mast, Jawanda; Riddell, Travis; Sepucha, Karen; Skavlem, Melissa; Skotko, Brian G.; Pediatrics, School of Medicine
    Purpose: We sought to determine if a novel online health tool, called Down Syndrome Clinic to You (DSC2U), could improve adherence to national Down syndrome (DS) guidelines. We also sought to determine if primary care providers (PCPs) and caregivers are satisfied with this personalized online health tool. Methods: In a national, randomized controlled trial of 230 caregivers who had children or dependents with DS without access to a DS specialist, 117 were randomized to receive DSC2U and 113 to receive usual care. The primary outcome was adherence to five health evaluations indicated by national guidelines for DS. DSC2U is completed electronically, in all mobile settings, by caregivers at home. The outputs-personalized checklists-are used during annual wellness visits with the patient's PCP. Results: A total of 213 participants completed a 7-month follow-up evaluation. In the intention-to-treat analysis, the intervention group had a 1.6-fold increase in the number of indicated evaluations that were recommended by the primary care provider or completed compared with controls. Both caregivers and PCPs reported high levels of satisfaction with DSC2U. Conclusions: DSC2U improved adherence to the national DS health-care guidelines with a novel modality that was highly valued by both caregivers and PCPs.
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    Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis
    (Wiley, 2023-09) Quintana, Melanie; Saville, Benjamin R.; Vestrucci, Matteo; Detry, Michelle A.; Chibnik, Lori; Shefner, Jeremy; Berry, James D.; Chase, Marianne; Andrews, Jinsy; Sherman, Alexander V.; Yu, Hong; Drake, Kristin; Cudkowicz, Merit; Paganoni, Sabrina; Macklin, Eric A.; HEALEY ALS Platform Trial Study Group; Physical Medicine and Rehabilitation, School of Medicine
    Platform trials allow efficient evaluation of multiple interventions for a specific disease. The HEALEY ALS Platform Trial is testing multiple investigational products in parallel and sequentially in persons with amyotrophic lateral sclerosis (ALS) with the goal of rapidly identifying novel treatments to slow disease progression. Platform trials have considerable operational and statistical efficiencies compared with typical randomized controlled trials due to their use of shared infrastructure and shared control data. We describe the statistical approaches required to achieve the objectives of a platform trial in the context of ALS. This includes following regulatory guidance for the disease area of interest and accounting for potential differences in outcomes of participants within the shared control (potentially due to differences in time of randomization, mode of administration, and eligibility criteria). Within the HEALEY ALS Platform Trial, the complex statistical objectives are met using a Bayesian shared parameter analysis of function and survival. This analysis serves to provide a common integrated estimate of treatment benefit, overall slowing in disease progression, as measured by function and survival while accounting for potential differences in the shared control group using Bayesian hierarchical modeling. Clinical trial simulation is used to provide a better understanding of this novel analysis method and complex design.